CHOC Using Stem Cells To Study Impact Of Autism

Last year, CHOC Children’s Research Institute received a National Institutes of Health (NIH) Research Grant to generate, investigate, and store neural stem cells derived from skin cells, donated by children with autism. The program is designed to provide an important new tool for studying the impact of autism on the developing brain.

Check out what Philip H. Schwartz, Ph.D., principal investigator on the NIH grant and founding director, National Human Neural Stem Cell Resource at CHOC, has to say about this exciting research:

Q: How unique is this study, and what do we hope to learn from it?A: By using easily obtainable skin cells, we can now generate patient-specific brain cells in the laboratory. This allows us to study what is going wrong in the brain of a patient with a genetic disease such as autism without ever having to touch their brain, a huge leap forward if there ever was one!

Only a very few laboratories are doing this and, in fact, the National Institutes of Health is convening a special meeting of scientists, including me, this October to discuss the best ways to move this new and exciting research forward.

Q: What are neural stem cells and how are they obtained?
A: Neural stem cells are immature brain cells that can divide many, many times and can mature into all the types of brain cells that make up our brains; all the brain cells that make up our entire brain are derived from neural stem cells. We can obtain these cells from the brain itself during surgery or after death or we can derive these cells, using modern technology, from skin rather than the brain.

Q: How will this study benefit patients and families?
A: Because we can now make brain cells from skin, we can now study brain cells from many patients simultaneously. This will allow us to directly probe what is wrong with these cells and, as a result, come up with new ways to diagnose and treat these very prevalent brain diseases.

Importantly, autism seems to be a class of diseases rather than a single disease and because we can now make patient-specific brain cells from the patient’s own skin, we may be able to tailor therapy to the patient.